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ML-driven target discovery

by insitroUS

ResearchStage 1 of 5

Discovery and lab stage; has picked ALS targets with a partner.

Updated Oct 2020Checked 25 Sep0 updates this week

Milestones

Next · First own drug into trials
  1. BMS ALS collaborationOct 2020Complete.
  2. First own drug into trialsNowCurrent milestone.

Most important updates

  • Oct 2020

Current obstacles

  • From dish to patientWhat happens in cells in a dish may not match the disease in people, a long-standing gap.

Physics limits

  • Cells in a dish are not a patientLab cells lack the tissues, immune system, blood flow and decades of disease history of a real body. Signals found in dishes often vanish in people, so trials remain the real test.
  • Diseases of decades don't fit in a dishIllnesses like ALS unfold over years, in aged tissue. Stem-cell-derived cells are young and simple, so they capture only early or partial versions of the disease.
  • Binding a target is not the same as a safe, working drugWhether a molecule is absorbed, reaches the right tissue, avoids toxicity and actually changes the disease depends on whole-body biology that no current model predicts. About 90% of drugs entering trials fail.

How it works

3 parts
Neurons (green) grown from stem cells in a dish (rat cells): insitro grows patient-derived stem cells into neurons to study disease
Neurons (green) grown from stem cells in a dish (rat cells): insitro grows patient-derived stem cells into neurons to study diseasePhoto: Yirui Sun · CC BY 4.0 (opens wellcomecollection.org)
Genetics

Start from human DNA

Large human datasets link gene variants to disease, pointing to causes rather than side effects.

Cells

Patient-like cells in a dish

Stem cells carrying those variants are grown into neurons or liver cells, so the disease can be studied in the relevant cell type.

Learn

AI finds the signal

Automated imaging and measurements feed models that pick out disease features and candidate targets for drugs.

Update log

1 update

Oct 2020

  • Minor: PressPartnership

About insitro

The team behind ML-driven target discovery

insitro

Machine learning, genetics, stem-cell models

South San Francisco company founded by Daphne Koller. It combines patient genetic data, lab-grown human cells and machine learning to find drug targets and predict who will respond, in brain and metabolic diseases.

  • Raised a $400M Series C in Mar 2021; partnered with Bristol Myers Squibb on ALS and Eli Lilly on metabolic disease.
  • Moving liver-targeted gene-silencing drugs for metabolic disease toward trials.
Founded
20188 yrs
Headquarters
United States
Status
Private
Valuation
private
Raised
$400M1 round
Last round
Series C · $400M15 Mar 2021
Works in
BiotechAI drug discovery
Coverage
1 program · 2 updateslatest 15 Mar 2021checked 25 Sep
Lead investors
CPP Investments
Primer